![]() ![]() ![]() ![]() 1 By introducing a certain nucleic acid modality to the desired tissue of the patient, gene expression can be downregulated, augmented or corrected. Gene therapy is a promising therapeutic platform because it targets disease-causing genes in a sequence-specific manner, which enables more precise and personalized treatment of diverse life-threatening diseases. This review provides a comprehensive view and roadmap for general readers working in the field. The latest progress in siRNA therapeutic development is also summarized. All clinically explored and commercialized siRNA delivery platforms, including the GalNAc ( N-acetylgalactosamine)–siRNA conjugate, and their fundamental design principles are thoroughly discussed. In this review, the evolution of siRNA chemical modifications and their biomedical performance are comprehensively reviewed. The drug development process was extremely hard, aiming to surmount complex obstacles, such as how to efficiently and safely deliver siRNAs to desired tissues and cells and how to enhance the performance of siRNAs with respect to their activity, stability, specificity and potential off-target effects. It can be administered quarterly, even twice-yearly, to achieve therapeutic effects, which is not the case for small molecules and antibodies. Reviewing the long-term pharmaceutical history of human beings, siRNA therapy currently has set up an extraordinary milestone, as it has already changed and will continue to change the treatment and management of human diseases. After a two-decade journey from its discovery, two approvals of siRNA therapeutics, ONPATTRO ® (patisiran) and GIVLAARI™ (givosiran), have been achieved by Alnylam Pharmaceuticals. It theoretically can silence any disease-related genes in a sequence-specific manner, making small interfering RNA (siRNA) a promising therapeutic modality. RNA interference (RNAi) is an ancient biological mechanism used to defend against external invasion. ![]()
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